The Food and Drug Administration on Thursday approved a drug Ionis Pharmaceuticals developed for the rare genetic disease hereditary angioedema, making the therapy, known as donidalorsen, the third new medicine to reach market this year for the rare genetic condition.
Donidalorsen, which Ionis will sell under the brand name Dawnzera, is approved to prevent the swelling attacks associated with hereditary angioedema in adults and children at least 12 years of age. Ionis will disclose the drug’s price on a conference call Thursday afternoon, a company spokesperson said.
HAE is a rare, inherited condition estimated to affect about 1 in every 50,000 people worldwide. The disease is characterized by recurrent swelling attacks that can last for days if untreated, and most commonly affect the limbs, face, gastrointestinal tract and throat. These episodes can be life-threatening.
Despite its rarity, HAE has become a crowded area of drug research. Prior to Dawnzera’s approval, several therapies were already available to treat or prevent these attacks. Two — Kalvista Pharmaceuticals’ Ekterly and CSL Behring’s Andembry — made it to market this year. A gene editing medicine is in late-stage testing, too.
As a result, investors’ expectations for Dawnzera are “relatively low,” analysts at the investment bank Stifel wrote in a client note in May.
Ionis, though, believes Dawnzera could become the preferred choice for patients looking to prevent swelling attacks. Current options, like the once-monthly injection Andembry and Biocryst Pharmaceuticals’ pill Orladeyo, reduce the rate of attacks, but don’t ward them off completely. In a poll Ionis conducted this year, a majority of patients contacted hadn’t yet found their best preventive option.
“Patients are looking for better efficacy, better convenience and better tolerability,” said CEO Brett Monia, in an interview. “At least one of those boxes are not checked with existing treatments,” he added, noting as evidence how people with HAE often “bounce around from one treatment to another” over the course of a year.
Dawnzera is different. It’s the first RNA-targeting medicine for HAE, and works by reducing levels of prekallikrein, a protein implicated in the onset of swelling attacks. Phase 3 results published in The New England Journal of Medicine last year showed that, among patients who received injections every four weeks, monthly attack rates were on average 81% lower than among those who received placebo. An every-other-month dose was associated with a 55% reduction versus placebo.
Monia also pointed to results from a study testing Dawnzera in patients who switched from another preventive therapy. That study found further reductions in swelling attack frequency among those who changed to Dawnzera. About 84% of trial participants also said they preferred Ionis’ treatment, citing convenience and better symptom management.
The most common side effects associated with treatment were injection site reactions, urinary and upper respiratory tract infections and abdominal discomfort.
“We don’t see any reason why [Dawnzera] would not be the preferred first-line treatment for those patients who are newly diagnosed,” Monia said. However, given the majority of patients are already taking preventive therapies, the company will focus its marketing efforts there, hoping the results it’s accrued will convince people to switch medications.
Ionis’ HAE drug is the second approved medicine the company will sell on its own. A pioneer in developing RNA medicines called antisense oligonucleotides, the company has long been known for partnering its products instead of commercializing them. But under Monia, who became CEO in 2020, the company has changed course.
Since shifting its strategy, the company has launched one drug on its own, Tryngolza, for a rare disorder called familial chylomicronemia syndrome. Multiple others are in late-stage testing.
“I felt it was going to be like turning around an aircraft carrier,” Monia said, but “the employees, the scientists, everybody at Ionis was ready for this.”
No comments:
Post a Comment
Note: Only a member of this blog may post a comment.